The invention provides methods of treating an obstructive coronary artery disease in a mammalian subject. The methods comprise administering to the subject an effective amount of an aromatic-cationic peptide to subjects in need thereof and performing a coronary artery bypass graft procedure on the subject.
The disclosure provides methods of preventing or treating heart failure in a mammalian subject, reducing risk factors associated with heart failure, and/or reducing the likelihood or severity of heart failure. The disclosure also provides methods of preventing, or treating LV remodeling in a mammalian subject, and/or reducing the likelihood or severity of LV remodeling. The methods comprise administering to the subject an effective amount of an aromatic-cationic peptide. In some embodiments, the methods comprise administering to the subject an effective amount of an aromatic cationic peptide to reduce levels of C-reactive protein, tumor necrosis factor alpha, interleukin 6, reactive oxygen species, Nt-pro BNP, and/or cardiac troponin I, and/or reduce expression levels of MLCL AT1 and/or ALCAT 1 in subjects in need thereof.
A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
C07K 5/11 - Tetrapeptides the side chain of the first amino acid containing more amino groups than carboxyl groups, or derivatives thereof, e.g. Lys, Arg
3.
CRYSTALLINE SALT FORMS OF BOC-D-ARG-DMT-LYS-(BOC)-PHE-NH2
C07K 5/11 - Tetrapeptides the side chain of the first amino acid containing more amino groups than carboxyl groups, or derivatives thereof, e.g. Lys, Arg
The disclosure provides methods of preventing or treating DMD in a mammalian subject, reducing risk factors associated with DMD, and/or reducing the likelihood or severity of DMD. The methods comprise administering to the subject an effective amount of an aromatic-cationic peptide.
The present disclosure provides methods, compounds, compositions/formulations/ medicaments and related uses for treating, preventing, delaying the progression of or delaying the onset of cardiomyopathy, such as hypertrophic cardiomyopathy, in a mammalian subject, such as a subject diagnosed with, or suspected or having, Friedreich's ataxia. In some embodiments, administration of the disclosed compounds to the subject may result in one or more of the following in the subject: (i) an increase in survival, (ii) reduction in iron deposits in cardiomyocytes, (iii) improvement and/or normalization of mitochondria size in cardiomyocytes; (iv) reduction in cardiac fibrosis, and (v) reverse remodeling of the subject's heart.
The present disclosure provides methods for treating, preventing, ameliorates, inhibits, or delaying the onset of Leigh syndrome as well as methods for ameliorating, inhibiting, ameliorates, inhibits, or delaying the onset of its associated signs and/or symptoms, such as exercise-induced acid acidosis, and increases in plasma levels of growth factor 15 (GDF-15) and/or fibroblast growth factor (FGF-21) in a subject in need thereof comprising administering to the subject various compounds, mixtures of compounds or compositions, formulations or medicaments derived therefrom.
A61P 7/08 - Plasma substitutesPerfusion solutionsDialytics or haemodialyticsDrugs for electrolytic or acid-base disorders, e.g. hypovolemic shock
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
05 - Pharmaceutical, veterinary and sanitary products
42 - Scientific, technological and industrial services, research and design
Goods & Services
Biopharmaceutical preparations for the treatment of mitochondrial, cardiovascular, renal, ophthalmic, neurologic and metabolic diseases and disorders; Pharmaceutical preparations for the prevention of mitochondrial, cardiovascular, renal, ophthalmic, neurologic and metabolic diseases and disorders; Biological preparations for the treatment of cellular abnormalities Scientific research and development; biopharmaceutical research, development and testing; Medical and scientific research in the field of mitochondrial, cardiovascular, renal, ophthalmic, neurologic, myopathic and metabolic systems
8.
CRYSTALLINE DIPEPTIDES USEFUL IN THE SYNTHESIS OF ELAMIPRETIDE
C07K 5/072 - Dipeptides the side chain of the first amino acid containing more carboxyl groups than amino groups, or derivatives thereof, e.g. Asp, Glu, Asn
C07C 237/20 - Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups having the carbon atoms of the carboxamide groups bound to acyclic carbon atoms of the carbon skeleton the carbon skeleton containing six-membered aromatic rings
C07K 5/068 - Dipeptides the side chain of the first amino acid containing more amino groups than carboxyl groups, or derivatives thereof, e.g. Lys, Arg
9.
METHODS FOR THE TREATMENT OF MITOCHONDRIAL DISEASE
The disclosure provides methods of preventing, ameliorating or treating disruption of mitochondrial function and symptoms thereof. The methods provide administering aromatic-cationic peptides in effective amounts to prevent, treat or ameliorate the disruption of mitochondrial oxidative phosphorylation in a cell such as that found in a subject suffering from, or predisposed to a mitochondrial disease or disorder. In some embodiments, the methods comprise administering to a subject suffering from, or at risk for a mitochondrial disease or disorder, an effective amount of an aromatic-cationic peptide to subjects in need thereof.
C07K 5/11 - Tetrapeptides the side chain of the first amino acid containing more amino groups than carboxyl groups, or derivatives thereof, e.g. Lys, Arg
A61K 9/00 - Medicinal preparations characterised by special physical form
A61K 9/127 - Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
C07K 5/068 - Dipeptides the side chain of the first amino acid containing more amino groups than carboxyl groups, or derivatives thereof, e.g. Lys, Arg
C07K 5/087 - Tripeptides the side chain of the first amino acid containing carbocyclic rings, e.g. Phe, Tyr
C07K 5/09 - Tripeptides the side chain of the first amino acid containing more amino groups than carboxyl groups, or derivatives thereof, e.g. Lys, Arg
05 - Pharmaceutical, veterinary and sanitary products
Goods & Services
Biopharmaceutical preparations for the treatment of mitochondrial, cardiovascular, renal, ophthalmic, neurologic and metabolic diseases and disorders; Pharmaceutical preparations for the prevention of mitochondrial, cardiovascular, renal, ophthalmic, neurologic and metabolic diseases and disorders; Biological preparations for the treatment of cellular abnormalities
11.
METHODS AND COMPOSITIONS FOR THE TREATMENT OF AGE-RELATED MACULAR DEGENERATION
The present technology provides methods and compositions for treating, preventing and/or delaying (slowing) the progression of age-related macular degeneration (AMD). In particular, the present technology relates to the use of elamipretide in effective amounts to treat, prevent and/or delay (slow) the progression of photoreceptor loss, AMD, GA secondary to AMD, non-exudative (dry) age-related macular degeneration, intermediate age-related macular degeneration, age-related macular degeneration with photoreceptor loss or non-exudative (dry) age-related macular degeneration with photoreceptor loss in mammalian subjects.
The present disclosure provides methods for treating, preventing, ameliorates, inhibits, or delaying the onset of Leigh syndrome as well as methods for ameliorating, inhibiting, ameliorates, inhibits, or delaying the onset of its associated signs and/or symptoms in a subject in need thereof comprising administering to the subject various compounds, mixtures of compounds or compositions, formulations or medicaments derived therefrom. The present disclosure further provides compositions, formulations or medicaments and related uses for the treatment, prevention ameliorating, inhibiting, or delaying the onset of Leigh syndrome in a subject and addressing its associated signs and/or symptoms.
The present disclosure provides novel methods for treating, preventing, ameliorating, inhibiting and/or delaying the onset of tauopathies. The methods comprise administering to the subject an effective amount of a small molecule peptidomimetic, such as (R)-2-amino-N—((S)-1-(((S)-5-amino-1-(3-benzyl-1,2,4-oxadiazol-5-yl)pentyl)amino)-3-(4-hydroxy-2,6-dimethylphenyl)-1-oxopropan-2-yl)-5-guanidinopentanamide, or a pharmaceutically acceptable salt, stereoisomer, tautomer, hydrate, and/or solvate thereof.
05 - Pharmaceutical, veterinary and sanitary products
Goods & Services
Biopharmaceutical preparations for the treatment of mitochondrial, cardiovascular, renal, ophthalmic, neurologic and metabolic diseases and disorders; Pharmaceutical preparations for the prevention of mitochondrial, cardiovascular, renal, ophthalmic, neurologic and metabolic diseases and disorders; Biological preparations for the treatment of cellular abnormalities.
05 - Pharmaceutical, veterinary and sanitary products
Goods & Services
Biopharmaceutical preparations for the treatment of mitochondrial, cardiovascular, renal, ophthalmic, neurologic and metabolic diseases and disorders; Pharmaceutical preparations for the prevention of mitochondrial, cardiovascular, renal, ophthalmic, neurologic and metabolic diseases and disorders; Biological preparations for the treatment of cellular abnormalities.
16.
METHODS AND COMPOSITIONS FOR THE TREATMENT OR PREVENTION OF MUSCULAR DYSTROPHY
The disclosure provides methods for treating muscular dystrophy (MD), such as Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD) in a mammalian subject. The methods comprise administering to the subject an effective amount of a peptide such as H-D-Arg-2,6-Dmt-Lys-PHe-NH2 (SEQ ID NO: 2) (a.k.a. MTP-131 or elamipretide), or a pharmaceutically acceptable salt, hydrate, solvate and/or tautomer thereof, in combination with at least one phosphorodiamidate morpholino oligomer (PMO) or peptide-conjugated PMO (PPMO). The methods are particularly useful for increasing dystrophin expression levels in subjects diagnosed with, and/or being treated for, MD in an amount greater than what would be possible by treatment with the PMO or PPMO alone.
A61K 31/675 - Phosphorus compounds having nitrogen as a ring hetero atom, e.g. pyridoxal phosphate
A61K 47/64 - Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
A61P 21/00 - Drugs for disorders of the muscular or neuromuscular system
17.
METHODS FOR ADDRESSING INJECTION SITE REACTIONS ASSOCIATED WITH THE ADMINISTRATION OF BEVEMIPRETIDE
The present disclosure provides methods for treating, preventing, ameliorating, inhibiting or delaying the onset of injection site reactions associated with the subcutaneous administration of bevemipretide, or a pharmaceutically acceptable salt thereof. The methods may involve administration of inhibitors of the MRGPRX2 receptor and/or inhibitors of mast cell degranulation. In some cases, the methods involve administration of an effective amount of a flavonoid, a coumarin, a phenol, a terpenoid, mometasone furoate, tacrolimus, quercetin, diphenhydramine and/or ice.
A61K 31/353 - 3,4-Dihydrobenzopyrans, e.g. chroman, catechin
A61K 31/138 - Aryloxyalkylamines, e.g. propranolol, tamoxifen, phenoxybenzamine
A61K 31/436 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system containing a six-membered ring having oxygen as a ring hetero atom, e.g. rapamycin
A61K 31/58 - Compounds containing cyclopenta[a]hydrophenanthrene ring systemsDerivatives thereof, e.g. steroids containing heterocyclic rings, e.g. danazol, stanozolol, pancuronium or digitogenin
A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
The disclosure provides compositions and methods relating to aromatic-cationic peptides. The methods comprise administering to the subject an effective amount of an aromatic-cationic peptide to subjects in need thereof. For example, the peptides may be administered to subjects in need of a mitochondrial-targeted antioxidant.
C07K 5/11 - Tetrapeptides the side chain of the first amino acid containing more amino groups than carboxyl groups, or derivatives thereof, e.g. Lys, Arg
C07K 5/065 - Dipeptides the side chain of the first amino acid containing carbocyclic rings, e.g. Phe, Tyr
C07K 5/068 - Dipeptides the side chain of the first amino acid containing more amino groups than carboxyl groups, or derivatives thereof, e.g. Lys, Arg
C07K 5/072 - Dipeptides the side chain of the first amino acid containing more carboxyl groups than amino groups, or derivatives thereof, e.g. Asp, Glu, Asn
C07K 5/087 - Tripeptides the side chain of the first amino acid containing carbocyclic rings, e.g. Phe, Tyr
C07K 5/09 - Tripeptides the side chain of the first amino acid containing more amino groups than carboxyl groups, or derivatives thereof, e.g. Lys, Arg
C07K 5/107 - Tetrapeptides the side chain of the first amino acid containing carbocyclic rings, e.g. Phe, Tyr
The present disclosure provides methods for treating, preventing, ameliorates, inhibits, or delaying the onset of Leigh syndrome as well as methods for ameliorating, inhibiting, ameliorates, inhibits, or delaying the onset of its associated signs and/or symptoms in a subject in need thereof comprising administering to the subject various compounds, mixtures of compounds or compositions, formulations or medicaments derived therefrom. The present disclosure further provides compositions, formulations or medicaments and related uses for the treatment, prevention ameliorating, inhibiting, or delaying the onset of Leigh syndrome in a subject and addressing its associated signs and/or symptoms.
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
20.
COMBINATION THERAPY FOR THE TREATMENT OF FRIEDREICH'S ATAXIA
The present disclosure provides a combination therapy for treating, preventing, inhibiting, ameliorating, or delaying the onset of Friedreich's ataxia in a subject in need thereof comprising administering to the subject both elamipretide and omaveloxolone or pharmaceutically acceptable salts, stereoisomers, mixtures of stereoisomers, tautomers, hydrates, and/or solvates thereof. The present disclosure further provides an embodiment of a combination product suitable for administration to said subject.
A61K 31/277 - NitrilesIsonitriles having a ring, e.g. verapamil
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
21.
COMPOSITIONS AND METHODS FOR THE TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS, PARKINSON’S DISEASE, PARKINSON’S DISEASE WITH DEMENTIA, DEMENTIA WITH LEWY BODIES, AND MULTIPLE SYSTEM ATROPHY
The present disclosure provides novel methods for treating or preventing amyotrophic lateral sclerosis (ALS), methods for delaying the onset of neurological symptoms associated with ALS, increasing survival in subjects afflicted with ALS, and attenuating the decline of muscle strength associated with ALS in a subject in need thereof. The present disclosure also provides methods for treating or preventing α-synucleinopathy or TDP-43 proteinopathy. The methods comprise administering to the subject an effective amount of a mitochondria-targeting peptidomimetic compound, such as (R)-2-amino-N-((S)-1-(((S)-5-amino-1-(3-benzyl-1,2,4-oxadiazol-5-yl)pentyl)amino)-3-(4-hydroxy-2,6-dimethylphenyl)-1-oxopropan-2-yl)-5-guanidinopentanamide, or a pharmaceutically acceptable salt, stereoisomer, tautomer, hydrate, and/or solvate thereof.
A61K 31/135 - Amines, e.g. amantadine having aromatic rings, e.g. methadone
A61K 31/197 - Carboxylic acids, e.g. valproic acid having an amino group the amino and the carboxyl groups being attached to the same acyclic carbon chain, e.g. gamma-aminobutyric acid [GABA], beta-alanine, epsilon-aminocaproic acid or pantothenic acid
A61K 31/4152 - 1,2-Diazoles having oxo groups directly attached to the heterocyclic ring, e.g. antipyrine, phenylbutazone, sulfinpyrazone
A61K 31/4178 - 1,3-Diazoles not condensed and containing further heterocyclic rings, e.g. pilocarpine, nitrofurantoin
A61K 31/4184 - 1,3-Diazoles condensed with carbocyclic rings, e.g. benzimidazoles
A61K 38/30 - Insulin-like growth factors, i.e. somatomedins, e.g. IGF-1, IGF-2
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
22.
COMBINATION THERAPY FOR THE TREATMENT OF FRIEDREICH'S ATAXIA
The present disclosure provides a combination therapy for treating, preventing, inhibiting, ameliorating, or delaying the onset of Friedreich's ataxia in a subject in need thereof comprising administering to the subject both elamipretide and omaveloxolone or pharmaceutically acceptable salts, stereoisomers, mixtures of stereoisomers, tautomers, hydrates, and/or solvates thereof. The present disclosure further provides an embodiment of a combination product suitable for administration to said subject.
The present technology provides methods and compositions for treating, preventing and/or delaying (slowing) the progression of age-related macular degeneration (AMD). In particular, the present technology relates to the use of elamipretide in effective amounts to treat, prevent and/or delay (slow) the progression of photoreceptor loss, AMD, GA secondary to AMD, non-exudative (dry) age-related macular degeneration, intermediate age-related macular degeneration, age-related macular degeneration with photoreceptor loss or non¬ exudative (dry) age-related macular degeneration with photoreceptor loss in mammalian subjects.
The disclosure provides therapeutic compounds, compositions (e.g., therapeutic agents or medicaments) and methods for preventing or treating mitochondrial disease such as Friedreich's ataxia in a mammalian subject, reducing risk factors, signs and/or symptoms associated with mitochondrial disease, such as Friedreich's ataxia, and/or reducing the likelihood or severity of mitochondrial disease such as Friedreich's ataxia. The disclosure further provides novel intermediates for the production of said therapeutic compositions. In some instances, the intermediates may themselves by therapeutic agents or prodrugs of therapeutic agents (e.g. reduced forms of the therapeutic compounds).
The disclosure provides therapeutic compositions (i.e., therapeutic agents) and methods of preventing or treating Friedreich's ataxia in a mammalian subject, reducing risk factors, signs and/or symptoms associated with Friedreich's ataxia (e.g., Complex I deficiency), and/or reducing the likelihood or severity of Friedreich's ataxia. The disclosure further provides novel intermediates for the production of said therapeutic compositions and related reduced versions of said therapeutic compositions, which reduce forms may also be used as therapeutic agents (or prodrugs of the therapeutic agent(s)).
C07D 305/10 - Heterocyclic compounds containing four-membered rings having one oxygen atom as the only ring hetero atoms not condensed with other rings having one or more double bonds between ring members or between ring members and non-ring members
C07C 49/835 - Ketones containing a keto group bound to a six-membered aromatic ring containing hydroxy groups having unsaturation outside an aromatic ring
26.
METHODS FOR ADDRESSING INJECTION SITE REACTIONS ASSOCIATED WITH THE ADMINISTRATION OF BEVEMIPRETIDE
The present disclosure provides methods for treating, preventing, ameliorating, inhibiting or delaying the onset of injection site reactions associated with the subcutaneous administration of bevemipretide, or a pharmaceutically acceptable salt thereof. The methods may involve administration of inhibitors of the MRGPRX2 receptor and/or inhibitors of mast cell degranulation. In some cases, the methods involve administration of an effective amount of a flavonoid, a coumarin, a phenol, a terpenoid, mometasone furoate, tacrolimus, quercetin, diphenhydramine and/or ice.
A61K 31/135 - Amines, e.g. amantadine having aromatic rings, e.g. methadone
A61K 31/352 - Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having six-membered rings with one oxygen as the only ring hetero atom condensed with carbocyclic rings, e.g. cannabinols, methantheline
The disclosure provides therapeutic compositions (i.e., therapeutic agents) and methods of preventing or treating Friedreich's ataxia in a mammalian subject, reducing risk factors, signs and/or symptoms associated with Friedreich's ataxia (e.g., Complex I deficiency), and/or reducing the likelihood or severity of Friedreich's ataxia. The disclosure further provides novel intermediates for the production of said therapeutic compositions and related reduced versions of said therapeutic compositions, which reduce forms may also be used as therapeutic agents (or prodrugs of the therapeutic agent(s)).
C07D 305/10 - Heterocyclic compounds containing four-membered rings having one oxygen atom as the only ring hetero atoms not condensed with other rings having one or more double bonds between ring members or between ring members and non-ring members
C07C 49/835 - Ketones containing a keto group bound to a six-membered aromatic ring containing hydroxy groups having unsaturation outside an aromatic ring
28.
METHODS AND COMPOSITIONS FOR PREVENTING OR TREATING DOMINANT OPTIC ATROPHY
The disclosure generally describes methods of preventing or treating dominant optic atrophy. The methods comprise administering an effective amount of an aromatic-cationic peptide to subjects in need thereof. The present technology relates generally to the treatment or prevention of Leber's hereditary optic neuropathy (LHON) or dominant optic atrophy (DOA) in mammals through administration of therapeutically effective amounts of aromatic-cationic peptides to subjects in need thereof. In one aspect, the present disclosure provides a method of treating or preventing dominant optic atrophy in a mammalian subject in need thereof, the method comprising administering to the subject a therapeutically effective amount of a peptide.
The present disclosure provides methods for treating, preventing, ameliorating, inhibiting or delaying the onset of injection site reactions associated with the subcutaneous administration of elamipretide, or a pharmaceutically acceptable salt thereof. The methods may involve administration of inhibitors of the MRGPRX2 receptor and/or inhibitors of mast cell degranulation. In some cases, the methods involve administration of an effective amount of a flavonoid, a coumarin, a phenol, or a terpenoid. Such methods may include applying mometasone furoate, tacrolimus, quercetin, diphenhydramine and/or ice to the elamipretide injection site or intended (injection site to treat, prevent, ameliorate, inhibit or delay the onset of said injection site reactions (ISRs). The present disclosure also provides methods for subcutaneously administering a 60 mg dose of elamipretide, or a pharmaceutically acceptable salt thereof, to a subject in need thereof.
A61K 31/352 - Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having six-membered rings with one oxygen as the only ring hetero atom condensed with carbocyclic rings, e.g. cannabinols, methantheline
A61K 31/58 - Compounds containing cyclopenta[a]hydrophenanthrene ring systemsDerivatives thereof, e.g. steroids containing heterocyclic rings, e.g. danazol, stanozolol, pancuronium or digitogenin
30.
COMPOUNDS, COMPOSITIONS AND METHODS FOR THE PREVENTION AND/OR TREATMENT OF VARIOUS MITOCHONDRIAL DISEASES OR DISORDERS, INCLUDING FRIEDREICH'S ATAXIA
The disclosure provides various new and existing compounds for use alone or as formulated in a composition (e.g., medicaments) and related methods and uses for treating, preventing, inhibiting, ameliorating or delaying the onset of a disease, disorder or condition associated with ferroptosis in a mammalian subject. Such ferroptosis related diseases, disorders or conditions can include: Friedreich's ataxia, Leigh syndrome, Leber's Hereditary Optic Neuropathy (LHON), (proliferative, non-proliferative, diabetic or hypertensive) retinopathy, refractory epilepsy, Parkinson's disease (PD), Alzheimer's disease (AD), Huntington's disease (HD), Amyotrophic Lateral Sclerosis (ALS), ischemic stroke, a cardiomyopathy (e.g. cardiac ischemia-reperfusion injury, myocardial infarction, Barth cardiomyopathy, hypertrophic cardiomyopathy or heart failure), renal injury, renal ischemia reperfusion injury or acute renal failure.
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
C07C 39/19 - Compounds having at least one hydroxy or O-metal group bound to a carbon atom of a six-membered aromatic ring monocyclic with unsaturation outside the aromatic ring containing carbon-to-carbon double bonds but no carbon-to-carbon triple bonds
C07C 50/28 - Quinones containing groups having oxygen atoms singly bound to carbon atoms with monocyclic quinoid structure
05 - Pharmaceutical, veterinary and sanitary products
42 - Scientific, technological and industrial services, research and design
Goods & Services
biological preparations for the prevention and treatment of cellular abnormalities.; Biopharmaceutical preparations for the prevention and treatment of mitochondrial, cardiovascular, renal, ophthalmic, neurologic, myopathic and metabolic diseases and disorders Scientific research and development; biopharmaceutical research, development and testing; medical and scientific research in the mitochondrial, cardiovascular, renal, ophthalmic, neurologic, myopathic and metabolic system fields.
05 - Pharmaceutical, veterinary and sanitary products
42 - Scientific, technological and industrial services, research and design
Goods & Services
Biopharmaceutical preparations for the prevention and treatment of mitochondrial, cardiovascular, renal, ophthalmic, neurologic, myopathic and metabolic diseases and disorders; biological preparations for the prevention and treatment of cellular abnormalities. Scientific research and development; biopharmaceutical research, development and testing; medical and scientific research in the mitochondrial, cardiovascular, renal, ophthalmic, neurologic, myopathic and metabolic system fields.
05 - Pharmaceutical, veterinary and sanitary products
42 - Scientific, technological and industrial services, research and design
Goods & Services
Biopharmaceutical preparations for the prevention and treatment of mitochondrial, cardiovascular, renal, ophthalmic, neurologic, myopathic and metabolic diseases and disorders; biological preparations for the prevention and treatment of cellular abnormalities. biopharmaceutical research, development and testing; medical and scientific research in the mitochondrial, cardiovascular, renal, ophthalmic, neurologic, myopathic and metabolic system fields.; Scientific research and development
05 - Pharmaceutical, veterinary and sanitary products
42 - Scientific, technological and industrial services, research and design
Goods & Services
biological preparations for the prevention and treatment of cellular abnormalities.; Biopharmaceutical preparations for the prevention and treatment of mitochondrial, cardiovascular, renal, ophthalmic, neurologic, myopathic and metabolic diseases and disorders biopharmaceutical research, development and testing; Scientific research and development; medical and scientific research in the mitochondrial, cardiovascular, renal, ophthalmic, neurologic, myopathic and metabolic system fields.
05 - Pharmaceutical, veterinary and sanitary products
42 - Scientific, technological and industrial services, research and design
Goods & Services
Biopharmaceutical preparations for the prevention and treatment of mitochondrial, cardiovascular, renal, ophthalmic, neurologic, myopathic and metabolic diseases and disorders; biological preparations for the prevention and treatment of cellular abnormalities. biopharmaceutical research, development and testing; Scientific research and development; medical and scientific research in the mitochondrial, cardiovascular, renal, ophthalmic, neurologic, myopathic and metabolic system fields.
36.
SILICA-BASED FORMULATIONS OF THERAPEUTIC OLIOGPEPTIDES AND PEPTIDOMIMETICS
Disclosed are depot formulations comprising a biodegradable silica hydrogel composite, wherein the hydrogel composite comprises (i) a silica hydrogel; and (ii) a plurality of microparticles comprising silica and an oligopeptide or a peptidomimetic compound, or a pharmaceutically acceptable salt thereof. The depot formulations described herein provide sustained, long-term release of the active pharmaceutical compound. The depot formulations may be implanted in the eye and are useful for treating ocular diseases. The plurality of microparticles comprising silica and an oligopeptide or a peptidomimetic compound, or a pharmaceutically acceptable salt thereof can be prepared using a continuous flow process.
A61K 41/17 - Inactivation or decontamination of a medicinal preparation prior to administration to an animal or a person by ultraviolet [UV] or infrared [IR] light, X-rays or gamma rays
A61M 5/32 - NeedlesDetails of needles pertaining to their connection with syringe or hubAccessories for bringing the needle into, or holding the needle on, the bodyDevices for protection of needles
37.
Methods and compositions for the prevention or treatment of Barth Syndrome
The disclosure provides methods of preventing or treating Barth Syndrome in a mammalian subject, reducing risk factors associated with Barth Syndrome, and/or reducing the likelihood or severity of Barth Syndrome. The methods comprise administering to the subject an effective amount of an aromatic-cationic peptide to increase expression of TAZ1 in subjects in need thereof.
05 - Pharmaceutical, veterinary and sanitary products
42 - Scientific, technological and industrial services, research and design
Goods & Services
Biopharmaceutical preparations for the prevention and treatment of mitochondrial, cardiovascular, renal, ophthalmic, neurologic, myopathic and metabolic diseases and disorders; biological preparations for the prevention and treatment of cellular abnormalities Scientific research and development; biopharmaceutical research, development and testing; medical and scientific research in the mitochondrial, cardiovascular, renal, ophthalmic, neurologic, myopathic and metabolic system field
39.
METHODS AND COMPOSITIONS FOR THE TREATMENT OF NEURODEGENERATIVE DISEASE
The present disclosure provides methods for achieving optimal levels of bevemipretide (also known as "(R)-2-amino-N-((S)- 1 -(((S)-5-amino- 1 -(3 -benzyl- 1,2,4-oxadiazol-5- yl)pentyl)amino)-3-(4-hydroxy-2,6-dimethylphenyl)-l-oxopropan-2-yl)-5- guanidinopentanamide" or "SBT-272"), or a pharmaceutically acceptable salt, stereoisomer, tautomer, hydrate, and/or solvate thereof, in brain tissue of subjects suspected of having, suffering from, or at risk for a neurodegenerative disease, such as, but not limited to amyotrophic lateral sclerosis (ALS), Parkinson's disease (PD), PD with dementia, dementia with Lewy bodies, Multiple System Atrophy, Huntington's disease, HTT proteinopathy, Frontotemporal Lobar Degeneration (FTLD), a tauopathy, and other disease where TDP-43, Tau protein, and α-synuclein are associated with the disease pathology.
A61K 31/135 - Amines, e.g. amantadine having aromatic rings, e.g. methadone
A61K 31/197 - Carboxylic acids, e.g. valproic acid having an amino group the amino and the carboxyl groups being attached to the same acyclic carbon chain, e.g. gamma-aminobutyric acid [GABA], beta-alanine, epsilon-aminocaproic acid or pantothenic acid
A61K 31/4152 - 1,2-Diazoles having oxo groups directly attached to the heterocyclic ring, e.g. antipyrine, phenylbutazone, sulfinpyrazone
A61K 31/4178 - 1,3-Diazoles not condensed and containing further heterocyclic rings, e.g. pilocarpine, nitrofurantoin
A61K 31/4184 - 1,3-Diazoles condensed with carbocyclic rings, e.g. benzimidazoles
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
C07K 5/11 - Tetrapeptides the side chain of the first amino acid containing more amino groups than carboxyl groups, or derivatives thereof, e.g. Lys, Arg
41.
THERAPEUTIC COMPOSITIONS INCLUDING SPN10 AND USES THEREOF
Disclosed herein are methods and compositions for the treatment, prevention, inhibition, amelioration and/or delay in the onset of diseases, disorders and/or conditions comprising administration of SPN10 or an analogue thereof, alone or in combination with one or more additional active agents (e.g. active pharmaceutical ingredients), wherein SPN10 is the aromatic-cationic peptide, H-L-Trp-L-Arg-L-Trp-L-Lys-NH2, or a pharmaceutically acceptable salt, tautomer, hydrate, and/or solvate thereof.
A61K 47/64 - Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
C07K 7/06 - Linear peptides containing only normal peptide links having 5 to 11 amino acids
42.
METHODS AND COMPOSITIONS FOR THE PREVENTION AND TREATMENT OF FRIEDREICH'S ATAXIA
The disclosure provides methods of preventing or treating Friedreich's ataxia in a mammalian subject, reducing risk factors, signs and/or symptoms associated with Friedreich's ataxia, and/or reducing the likelihood or severity of Friedreich's ataxia. The methods comprise administering to the subject an effective amount of an aromatic-cationic peptide to e.g., reduce oxidative stress, increase mitochondrial metabolism, or a combination thereof.
A61K 31/122 - Ketones having the oxygen atom directly attached to a ring, e.g. quinones, vitamin K1, anthralin
A61K 31/401 - ProlineDerivatives thereof, e.g. captopril
A61K 31/4412 - Non-condensed pyridinesHydrogenated derivatives thereof having oxo groups directly attached to the heterocyclic ring
A61K 31/7048 - Compounds having saccharide radicals and heterocyclic rings having oxygen as a ring hetero atom, e.g. leucoglucosan, hesperidin, erythromycin, nystatin
A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
C07K 5/11 - Tetrapeptides the side chain of the first amino acid containing more amino groups than carboxyl groups, or derivatives thereof, e.g. Lys, Arg
Disclosed are deuterated analogs of SBT-20 and elamipretide (MTP-131). The compounds are useful for the treatment and prevention of ischemia-reperfusion injury (e.g., cardiac ischemia-reperfusion injury) or myocardial infarction.
Disclosed are non-natural peptides useful for the treatment and prevention of ischemia-reperfusion injury (e.g., cardiac ischemia-reperfusion injury) or myocardial infarction.
C07K 5/09 - Tripeptides the side chain of the first amino acid containing more amino groups than carboxyl groups, or derivatives thereof, e.g. Lys, Arg
C07K 5/072 - Dipeptides the side chain of the first amino acid containing more carboxyl groups than amino groups, or derivatives thereof, e.g. Asp, Glu, Asn
C07D 413/06 - Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms containing two hetero rings linked by a carbon chain containing only aliphatic carbon atoms
45.
COMPOSITIONS AND METHODS FOR THE PREVENTION AND TREATMENT OF MITOCHONDRIAL MYOPATHIES
The disclosure provides methods of preventing or treating mitochondrial myopathy in a mammalian subject, reducing risk factors associated with mitochondrial myopathy, and/or reducing the likelihood or severity of mitochondrial myopathy. The methods comprise administering to the subject an effective amount of an aromatic-cationic peptide.
C07K 5/11 - Tetrapeptides the side chain of the first amino acid containing more amino groups than carboxyl groups, or derivatives thereof, e.g. Lys, Arg
The disclosure generally describes methods of preventing or treating Lebers hereditary optic neuropathy (LHON). The methods comprise administering an effective amount of an aromatic-cationic peptide to subjects in need thereof.
The disclosure generally relates to compounds (i.e. peptidomimetics), compositions (e.g. formulations or medicaments), methods and related uses for treating, preventing, inhibiting, amelioration or delaying the onset of ophthalmic diseases, disorders or conditions in a mammalian subject, such as a human. In some embodiments, the ophthalmic disease, disorder or condition may be associated with deterioration of the integrity of the ellipsoid zone of one or more eyes of the mammalian subject. The methods and uses comprise administering an effective amount of peptidomimetic (alone, as formulated and/or in combination with at least one additional therapeutic agent) to mammalian subjects in need thereof.
The disclosure generally describes methods of preventing or treating ophthalmic diseases or conditions in a mammalian subject, such as diabetic retinopathy, cataracts, retinitis pigmentosa, glaucoma, macular degeneration, choroidal neovascularization, retinal degeneration, and oxygen-induced retinopathy. The methods comprise administering an effective amount of an aromatic-cationic peptide to subjects in need thereof.
C07K 5/09 - Tripeptides the side chain of the first amino acid containing more amino groups than carboxyl groups, or derivatives thereof, e.g. Lys, Arg
C07K 5/107 - Tetrapeptides the side chain of the first amino acid containing carbocyclic rings, e.g. Phe, Tyr
49.
ADDRESSING INJECTION SITE REACTIONS ASSOCIATED WITH THE ADMINISTRATION OF ELAMIPRETIDE
The present disclosure provides methods for treating, preventing, ameliorating, inhibiting or delaying the onset of injection site reactions associated with the subcutaneous administration of elamipretide, or a pharmaceutically acceptable salt thereof. The methods may involve administration of inhibitors of the MRGPRX2 receptor and/or inhibitors of mast cell degranulation. In some cases, the methods involve administration of an effective amount of a flavonoid, a coumarin, a phenol, or a terpenoid. Such methods may include applying mometasone furoate, tacrolimus, quercetin, diphenhydramine and/or ice to the elamipretide injection site or intended injection site to treat, prevent, ameliorate, inhibit or delay the onset of said injection site reactions (ISRs). The present disclosure also provides methods for subcutaneously administering a 60mg dose of elamipretide, or a pharmaceutically acceptable salt thereof, to a subject in need thereof.
The present disclosure provides compounds, compositions, and/or methods for treating, preventing, inhibiting, ameliorating, or delaying the onset of Huntington's disease and/or a HTT proteinopathy in a subject. The methods can comprise administering to the subject an effective amount of a peptidomimetic compound, such as (R)-2-amino-N-((S)-l- (((S)-5-amino- 1 -(3 -benzyl- 1,2,4-oxadiazol-5-yl)pentyl)amino)-3 -(4-hydroxy-2,6- dimethylphenyl)-l-oxopropan-2-yl)-5-guanidinopentanamide, or a pharmaceutically acceptable salt, stereoisomer, tautomer, hydrate, and/or solvate thereof, alone or in combination with one or more other therapeutic agents.
The disclosure generally describes methods of preventing or treating ophthalmic diseases or conditions in a mammalian subject, such as diabetic retinopathy, cataracts, retinitis pigmentosa, glaucoma, macular degeneration, choroidal neovascularization, retinal degeneration, and oxygen-induced retinopathy. The methods comprise administering an effective amount of an aromatic-cationic peptide to subjects in need thereof.
C07K 5/09 - Tripeptides the side chain of the first amino acid containing more amino groups than carboxyl groups, or derivatives thereof, e.g. Lys, Arg
The disclosure provides therapeutic compositions (i.e., therapeutic agents) and methods of preventing or treating Friedreich's ataxia in a mammalian subject, reducing risk factors, signs and/or symptoms associated with Friedreich's ataxia (e.g., Complex I deficiency), and/or reducing the likelihood or severity of Friedreich's ataxia. The disclosure further provides novel intermediates for the production of said therapeutic compositions and related reduced versions of said therapeutic compositions, which reduce forms may also be used as therapeutic agents (or prodrugs of the therapeutic agent(s)).
C07D 305/10 - Heterocyclic compounds containing four-membered rings having one oxygen atom as the only ring hetero atoms not condensed with other rings having one or more double bonds between ring members or between ring members and non-ring members
C07C 49/835 - Ketones containing a keto group bound to a six-membered aromatic ring containing hydroxy groups having unsaturation outside an aromatic ring
The disclosure provides methods of preventing, ameliorating or treating disruption of mitochondrial function and symptoms thereof. The methods provide administering aromatic-cationic peptides in effective amounts to prevent, treat or ameliorate the disruption of mitochondrial oxidative phosphorylation in a cell such as that found in a subject suffering from, or predisposed to a mitochondrial disease or disorder. In some embodiments, the methods comprise administering to a subject suffering from, or at risk for a mitochondrial disease or disorder, an effective amount of an aromatic-cationic peptide to subjects in need thereof.
C07K 5/11 - Tetrapeptides the side chain of the first amino acid containing more amino groups than carboxyl groups, or derivatives thereof, e.g. Lys, Arg
C07K 5/09 - Tripeptides the side chain of the first amino acid containing more amino groups than carboxyl groups, or derivatives thereof, e.g. Lys, Arg
The disclosure provides methods for treating muscular dystrophy (MD), such as Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD) in a mammalian subject. The methods are particularly useful for treating, inhibiting, reducing, ameliorating or delaying the onset of hypertrophic cardiomyopathy, dilated cardiomyopathy, heart failure and/or cardiac fibrosis in subjects diagnosed with, and/or being treated for, MD.The methods comprise administering to the subject an effective amount of a peptide such as H-D-Arg-2,6-Dmt-Lys-PHe-NH2 (a.k.a. elamipretide), or a pharmaceutically acceptable salt, hydrate, solvate and/or tautomer thereof, optionally in combination with at least one other active ingredient (i.e. drug) such a corticosteroid, ACE inhibitor, ARB(s), beta-blocker or drug that increases or corrects the expression of dystrophin in the subject (e.g. Eteplirsen (Exondys 51®), Golodirsen (Vyondys 53™)) or Casimersen (Amondys 45™).
C07K 5/11 - Tetrapeptides the side chain of the first amino acid containing more amino groups than carboxyl groups, or derivatives thereof, e.g. Lys, Arg
C07K 5/107 - Tetrapeptides the side chain of the first amino acid containing carbocyclic rings, e.g. Phe, Tyr
59.
SMALL MOLECULE PEPTIDOMIMETIC FOR THE TREATMENT OF TAUOPATHIES
The present disclosure provides novel methods for treating, preventing, ameliorating, inhibiting and/or delaying the onset of tauopathies. The methods comprise administering to the subject an effective amount of a small molecule peptidomimetic, such as (R)-2-amino-N- ((S)-l-(((S)-5-amino-l-(3-benzyl-l,2,4-oxadiazol-5-yl)pentyl)amino)-3-(4-hydroxy-2,6- dimethylphenyl)-l-oxopropan-2-yl)-5-guanidinopentanamide, or a pharmaceutically acceptable salt, stereoisomer, tautomer, hydrate, and/or solvate thereof.
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
60.
Crystalline dipeptides useful in the synthesis of elamipretide
C07K 5/072 - Dipeptides the side chain of the first amino acid containing more carboxyl groups than amino groups, or derivatives thereof, e.g. Asp, Glu, Asn
C07C 237/20 - Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups having the carbon atoms of the carboxamide groups bound to acyclic carbon atoms of the carbon skeleton the carbon skeleton containing six-membered aromatic rings
C07K 5/068 - Dipeptides the side chain of the first amino acid containing more amino groups than carboxyl groups, or derivatives thereof, e.g. Lys, Arg
61.
METHODS FOR REDUCING RISKS ASSOCIATED WITH HEART FAILURE AND FACTORS ASSOCIATED THEREWITH
The disclosure provides methods of preventing or treating heart failure in a mammalian subject, reducing risk factors associated with heart failure, and/or reducing the likelihood or seventy of heart failure. The disclosure also provides methods of preventing, or treating LV remodeling in a mammalian subject, and/or reducing the likelihood or severity of LV remodeling. The methods comprise administering to the subject an effective amount of an aromatic-cationic peptide. In some embodiments, the methods comprise administering to the subject an effective amount of an aromatic cationic peptide to reduce levels of C-reactive protein, tumor necrosis factor alpha, interleukin 6, reactive oxygen species, Nt-pro BNP, and/or cardiac troponin I, and/or reduce expression levels of MLCL AT1 and/or ALCAT 1 in subjects in need thereof.
A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
C07K 5/11 - Tetrapeptides the side chain of the first amino acid containing more amino groups than carboxyl groups, or derivatives thereof, e.g. Lys, Arg
62.
METHODS AND COMPOSITIONS FOR THE TREATMENT OR PREVENTION OF MUSCULAR DYSTROPHY
The disclosure provides methods for treating muscular dystrophy (MD), such as Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD) in a mammalian subject. The methods comprise administering to the subject an effective amount of a peptide such as H-D-Arg-2,6-Dmt-Lys-PHe-NH2 (a.k.a. MTP-131 or elamipretide), or a pharmaceutically acceptable salt, hydrate, solvate and/or tautomer thereof, in combination with at least one phosphorodiamidate morpholino oligomer (PMO) or peptide-conjugated PMO (PPMO). The methods are particularly useful for increasing dystrophin expression levels in subjects diagnosed with, and/or being treated for, MD in an amount greater than what would be possible by treatment with the PMO or PPMO alone.
A61P 21/00 - Drugs for disorders of the muscular or neuromuscular system
C07K 5/11 - Tetrapeptides the side chain of the first amino acid containing more amino groups than carboxyl groups, or derivatives thereof, e.g. Lys, Arg
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
Disclosed are non-natural peptides useful for the treatment and prevention of ischemia-reperfusion injury (e.g., cardiac ischemia-reperfusion injury) or myocardial infarction.
C07K 5/09 - Tripeptides the side chain of the first amino acid containing more amino groups than carboxyl groups, or derivatives thereof, e.g. Lys, Arg
C07K 5/072 - Dipeptides the side chain of the first amino acid containing more carboxyl groups than amino groups, or derivatives thereof, e.g. Asp, Glu, Asn
C07D 413/06 - Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms containing two hetero rings linked by a carbon chain containing only aliphatic carbon atoms
64.
METHODS AND COMPOSITIONS COMPRISING PEPTIDOMIMITICS FOR TREATING, PREVENTING, INHIBITING, AMELIORATING OR DELAYING THE ONSET OF OPHTHALMIC CONDITIONS
The disclosure generally relates to compounds (j.e. peptidomimetics), compositions (e.g. formulations or medicaments), methods and related uses for treating, preventing, inhibiting, amelioration or delaying the onset of ophthalmic diseases, disorders or conditions in a mammalian subject, such as a human. In some embodiments, the ophthalmic disease, disorder or condition may be associated with deterioration of the integrity of the ellipsoid zone of one or more eyes of the mammalian subject. The methods and uses comprise administering an effective amount of peptidomimetic (alone, as formulated and/or in combination with at least one additional therapeutic agent) to mammalian subjects in need thereof.
The disclosure generally relates to compounds (j.e. peptidomimetics), compositions (e.g. formulations or medicaments), methods and related uses for treating, preventing, inhibiting, amelioration or delaying the onset of ophthalmic diseases, disorders or conditions in a mammalian subject, such as a human. In some embodiments, the ophthalmic disease, disorder or condition may be associated with deterioration of the integrity of the ellipsoid zone of one or more eyes of the mammalian subject. The methods and uses comprise administering an effective amount of peptidomimetic (alone, as formulated and/or in combination with at least one additional therapeutic agent) to mammalian subjects in need thereof.
The disclosure provides methods of preventing or treating DMD in a mammalian subject, reducing risk factors associated with DMD, and/or reducing the likelihood or severity of DMD. The methods comprise administering to the subject an effective amount of an aromatic-cationic peptide.
The present technology is directed to compositions and methods for preventing, ameliorating, or reducing the severity of one or more signs, or symptoms associated with a reduction of function, decreased expression level of, and/or deficiency in one or more of COL4A3, COL4A4 and COL4A5 genes. Also disclosed herein are methods of preventing or treating Alport Syndrome in a mammalian subject, reducing risk factors associated with Alport Syndrome, and/or reducing the likelihood or severity of Alport Syndrome. The methods comprise administering to the subject an effective amount of an aromatic-cationic peptide.
The disclosure provides methods for detecting and diagnosing diseases and conditions associated with defects in cardiolipin remodeling. In some embodiments, the present technology relates to methods for detecting the presence or amount of cardiolipin isoforms and/or the presence or amount of enzymes involved in cardiolipin remodeling.
C12Q 1/68 - Measuring or testing processes involving enzymes, nucleic acids or microorganismsCompositions thereforProcesses of preparing such compositions involving nucleic acids
C12Q 1/6883 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
The disclosure provides various new and existing compounds for use alone or as formulated in a composition (e.g., medicaments) and related methods and uses for treating, preventing, inhibiting, ameliorating or delaying the onset of a disease, disorder or condition associated with ferroptosis in a mammalian subject. Such ferroptosis related diseases, disorders or conditions can include: Friedreich's ataxia, Leigh syndrome, Leber's Hereditary Optic Neuropathy (LHON), (proliferative, non-proliferative, diabetic or hypertensive) retinopathy, refractory epilepsy, Parkinson's disease (PD), Alzheimer's disease (AD), Huntington's disease (HD), Amyotrophic Lateral Sclerosis (ALS), ischemic stroke, a cardiomyopathy (e.g. cardiac ischemia-reperfusion injury, myocardial infarction, Barth cardiomyopathy, hypertrophic cardiomyopathy or heart failure), renal injury, renal ischemia reperfusion injury or acute renal failure.
Disclosed are depot formulations comprising a biodegradable silica hydrogel composite, wherein the hydrogel composite comprises (i) a silica hydrogel; and (ii) a plurality of microparticles comprising silica and an oligopeptide or a peptidomimetic compound, or a pharmaceutically acceptable salt thereof. The depot formulations described herein provide sustained, long-term release of the active pharmaceutical compound. The depot formulations may be implanted in the eye and are useful for treating ocular diseases. The plurality of microparticles comprising silica and an oligopeptide or a peptidomimetic compound, or a pharmaceutically acceptable salt thereof can be prepared using a continuous flow process.
The disclosure relates to methods for treating a subject suffering from hyperalgesia caused by drug-induced neuropathy by administering to the subject an effective amount of an aromatic-cationic peptide. The disclosure also relates to methods for protecting a subject from hyperalgesia caused by drug-induced neuropathy by administering an effective amount of an aromatic-cationic peptide to a subject at risk for developing the condition.
A61K 31/4748 - QuinolinesIsoquinolines forming part of bridged ring systems
A61K 31/55 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having seven-membered rings, e.g. azelastine, pentylenetetrazole
A61K 31/7042 - Compounds having saccharide radicals and heterocyclic rings
The disclosure provides methods of preventing, treating, or ameliorating LV remodeling in a mammalian subject. The methods comprise administering to the subject a therapeutic amount of an aromatic-cationic peptide such as D-Arg-2,6-Dmt-Lys-Phe-NH2.
A61K 9/00 - Medicinal preparations characterised by special physical form
C07K 5/09 - Tripeptides the side chain of the first amino acid containing more amino groups than carboxyl groups, or derivatives thereof, e.g. Lys, Arg
73.
METHODS FOR PERFORMING A CORONARY ARTERY BYPASS GRAFT PROCEDURE
The invention provides methods of treating an obstructive coronary artery disease in a mammalian subject. The methods comprise administering to the subject an effective amount of an aromatic-cationic peptide to subjects in need thereof and performing a coronary artery bypass graft procedure on the subject.
A61M 39/08 - TubesStorage means specially adapted therefor
74.
Compositions and methods for the treatment of amyotrophic lateral sclerosis, parkinson's disease, parkinson's disease with dementia, dementia with lewy bodies, and multiple system atrophy
The present disclosure provides novel methods for treating or preventing amyotrophic lateral sclerosis (ALS), methods for delaying the onset of neurological symptoms associated with ALS, increasing survival in subjects afflicted with ALS, and attenuating the decline of muscle strength associated with ALS in a subject in need thereof. The present disclosure also provides methods for treating or preventing α-synucleinopathy or TDP-43 proteinopathy. The methods comprise administering to the subject an effective amount of a mitochondria-targeting peptidomimetic compound, such as (R)-2-amino-N—((S)-1-(((S)-5-amino-1-(3-benzyl-1,2,4-oxadiazol-5-yl)pentyl)amino)-3-(4-hydroxy-2,6-dimethylphenyl)-1-oxopropan-2-yl)-5-guanidinopentanamide, or a pharmaceutically acceptable salt, stereoisomer, tautomer, hydrate, and/or solvate thereof.
A61K 31/135 - Amines, e.g. amantadine having aromatic rings, e.g. methadone
A61K 31/197 - Carboxylic acids, e.g. valproic acid having an amino group the amino and the carboxyl groups being attached to the same acyclic carbon chain, e.g. gamma-aminobutyric acid [GABA], beta-alanine, epsilon-aminocaproic acid or pantothenic acid
A61K 31/4152 - 1,2-Diazoles having oxo groups directly attached to the heterocyclic ring, e.g. antipyrine, phenylbutazone, sulfinpyrazone
A61K 31/4178 - 1,3-Diazoles not condensed and containing further heterocyclic rings, e.g. pilocarpine, nitrofurantoin
A61K 31/4184 - 1,3-Diazoles condensed with carbocyclic rings, e.g. benzimidazoles
A61K 38/30 - Insulin-like growth factors, i.e. somatomedins, e.g. IGF-1, IGF-2
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
05 - Pharmaceutical, veterinary and sanitary products
42 - Scientific, technological and industrial services, research and design
Goods & Services
Biopharmaceutical preparations for the prevention and treatment of mitochondrial, cardiovascular, renal, ophthalmic, neurologic, myopathic and metabolic diseases and disorders; biological preparations for the prevention and treatment of cellular abnormalities Scientific research and development; biopharmaceutical research, development and testing; medical and scientific research in the mitochondrial, cardiovascular, renal, ophthalmic, neurologic, myopathic and metabolic system fields
Disclosed are analogs of SBT-20. The compounds are useful for the treatment and prevention of ischemia-reperfusion injury (e.g., cardiac ischemia-reperfusion injury) or myocardial infarction.
C07K 5/107 - Tetrapeptides the side chain of the first amino acid containing carbocyclic rings, e.g. Phe, Tyr
A61P 9/10 - Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
C07K 7/06 - Linear peptides containing only normal peptide links having 5 to 11 amino acids
Disclosed are analogs of elamipretide (MTP-131). The compounds are useful for the treatment and prevention of ischemia-reperfusion injury (e.g., cardiac ischemia-reperfusion injury) or myocardial infarction.
C07K 5/11 - Tetrapeptides the side chain of the first amino acid containing more amino groups than carboxyl groups, or derivatives thereof, e.g. Lys, Arg
C07K 5/117 - Tetrapeptides the first amino acid being heterocyclic, e.g. Pro, His, Trp
78.
Methods and compositions for the treatment of Sengers syndrome
The disclosure provides methods of preventing or treating Sengers syndrome in a mammalian subject, reducing risk factors associated with Sengers syndrome, and/or reducing the likelihood or severity of Sengers syndrome. The methods comprise administering to the subject an effective amount of an aromatic-cationic peptide to increase expression of AGK in subjects in need thereof.
The disclosure provides methods of preventing or treating Barth Syndrome in a mammalian subject, reducing risk factors associated with Barth Syndrome, and/or reducing the likelihood or severity of Barth Syndrome. The methods comprise administering to the subject an effective amount of an aromatic-cationic peptide to increase expression of TAZ1 in subjects in need thereof.
2 2 (a.k.a. elamipretide), or a pharmaceutically acceptable salt, hydrate, solvate and/or tautomer thereof, optionally in combination with at least one other active ingredient (i.e. drug) such a corticosteroid, ACE inhibitor, ARB(s), beta-blocker or drug that increases or corrects the expression of dystrophin in the subject (e.g. Eteplirsen (Exondys 51®), Golodirsen (Vyondys 53™)) or Casimersen (Amondys 45™).
C07K 5/11 - Tetrapeptides the side chain of the first amino acid containing more amino groups than carboxyl groups, or derivatives thereof, e.g. Lys, Arg
A61K 31/513 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim having oxo groups directly attached to the heterocyclic ring, e.g. cytosine
C07D 401/12 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing two hetero rings linked by a chain containing hetero atoms as chain links
81.
Compositions and methods for the prevention and/or treatment of mitochondrial disease, including Friedreich's ataxia
The disclosure provides therapeutic compounds, compositions (e.g., therapeutic agents or medicaments) and methods for preventing or treating mitochondrial disease such as Friedreich's ataxia in a mammalian subject, reducing risk factors, signs and/or symptoms associated with mitochondrial disease, such as Friedreich's ataxia, and/or reducing the likelihood or severity of mitochondrial disease such as Friedreich's ataxia. The disclosure further provides novel intermediates for the production of said therapeutic compositions. In some instances, the intermediates may themselves by therapeutic agents or prodrugs of therapeutic agents (e.g. reduced forms of the therapeutic compounds).
C07K 5/09 - Tripeptides the side chain of the first amino acid containing more amino groups than carboxyl groups, or derivatives thereof, e.g. Lys, Arg
C07K 5/107 - Tetrapeptides the side chain of the first amino acid containing carbocyclic rings, e.g. Phe, Tyr
83.
Crystalline dipeptides useful in the synthesis of elamipretide
C07K 5/072 - Dipeptides the side chain of the first amino acid containing more carboxyl groups than amino groups, or derivatives thereof, e.g. Asp, Glu, Asn
C07K 5/068 - Dipeptides the side chain of the first amino acid containing more amino groups than carboxyl groups, or derivatives thereof, e.g. Lys, Arg
C07C 237/20 - Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups having the carbon atoms of the carboxamide groups bound to acyclic carbon atoms of the carbon skeleton the carbon skeleton containing six-membered aromatic rings
C07K 5/11 - Tetrapeptides the side chain of the first amino acid containing more amino groups than carboxyl groups, or derivatives thereof, e.g. Lys, Arg
05 - Pharmaceutical, veterinary and sanitary products
Goods & Services
Biopharmaceutical preparations for the prevention and treatment of cardiovascular, renal, ophthalmic, neurologic and metabolic diseases and disorders; biological preparations for the prevention and treatment of cellular abnormalities
05 - Pharmaceutical, veterinary and sanitary products
Goods & Services
Biopharmaceutical preparations for the prevention and treatment of cardiovascular, renal, ophthalmic, neurologic and metabolic diseases and disorders; biological preparations for the prevention and treatment of cellular abnormalities
87.
Methods for treating heart disease in a subject with Friedreich's ataxia by an aromatic-cationic peptide
The disclosure provides methods of preventing or treating Friedreich's ataxia in a mammalian subject, reducing risk factors, signs and/or symptoms associated with Friedreich's ataxia, and/or reducing the likelihood or severity of Friedreich's ataxia. The methods comprise administering to the subject an effective amount of an aromatic-cationic peptide to e.g., reduce oxidative stress, increase mitochondrial metabolism, or a combination thereof.
A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
A61K 31/122 - Ketones having the oxygen atom directly attached to a ring, e.g. quinones, vitamin K1, anthralin
A61K 31/401 - ProlineDerivatives thereof, e.g. captopril
A61K 31/4412 - Non-condensed pyridinesHydrogenated derivatives thereof having oxo groups directly attached to the heterocyclic ring
A61K 31/7048 - Compounds having saccharide radicals and heterocyclic rings having oxygen as a ring hetero atom, e.g. leucoglucosan, hesperidin, erythromycin, nystatin
C07K 5/11 - Tetrapeptides the side chain of the first amino acid containing more amino groups than carboxyl groups, or derivatives thereof, e.g. Lys, Arg
A61P 43/00 - Drugs for specific purposes, not provided for in groups
A61P 21/00 - Drugs for disorders of the muscular or neuromuscular system
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
88.
Therapeutic compositions including peptides and uses thereof
Disclosed herein are methods and compositions for the treatment and/or prevention of ischemia and ischemia-reperfusion injury comprising administration of at least one peptide disclosed herein, or a pharmaceutically acceptable salt thereof.
C07K 7/06 - Linear peptides containing only normal peptide links having 5 to 11 amino acids
A61P 9/10 - Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
The disclosure provides therapeutic compositions (i.e., therapeutic agents) and methods of preventing or treating Friedreich's ataxia in a mammalian subject, reducing risk factors, signs and/or symptoms associated with Friedreich's ataxia (e.g., Complex I deficiency), and/or reducing the likelihood or severity of Friedreich's ataxia. The disclosure further provides novel intermediates for the production of said therapeutic compositions and related reduced versions of said therapeutic compositions, which reduce forms may also be used as therapeutic agents (or prodrugs of the therapeutic agent(s)).
C07C 49/835 - Ketones containing a keto group bound to a six-membered aromatic ring containing hydroxy groups having unsaturation outside an aromatic ring
C07D 305/10 - Heterocyclic compounds containing four-membered rings having one oxygen atom as the only ring hetero atoms not condensed with other rings having one or more double bonds between ring members or between ring members and non-ring members
05 - Pharmaceutical, veterinary and sanitary products
42 - Scientific, technological and industrial services, research and design
Goods & Services
Biopharmaceutical preparations for the prevention and treatment of mitochondrial, cardiovascular, renal, ophthalmic, neurologic, myopathic and metabolic diseases and disorders; biological preparations for the prevention and treatment of cellular abnormalities Scientific research and development; biopharmaceutical research, development and testing; medical and scientific research in the mitochondrial, cardiovascular, renal, ophthalmic, neurologic, myopathic and metabolic system fields
05 - Pharmaceutical, veterinary and sanitary products
42 - Scientific, technological and industrial services, research and design
Goods & Services
Biopharmaceutical preparations for the prevention and treatment of mitochondrial, cardiovascular, renal, ophthalmic, neurologic, myopathic and metabolic diseases and disorders; biological preparations for the prevention and treatment of cellular abnormalities Scientific research and development; biopharmaceutical research, development and testing; medical and scientific research in the mitochondrial, cardiovascular, renal, ophthalmic, neurologic, myopathic and metabolic system fields
92.
Methods and compositions for preventing or treating ophthalmic conditions
The disclosure generally describes methods of preventing or treating ophthalmic diseases or conditions in a mammalian subject, such as diabetic retinopathy, cataracts, retinitis pigmentosa, glaucoma, macular degeneration, choroidal neovascularization, retinal degeneration, and oxygen-induced retinopathy. The methods comprise administering an effective amount of an aromatic-cationic peptide to subjects in need thereof.
A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
C07K 5/09 - Tripeptides the side chain of the first amino acid containing more amino groups than carboxyl groups, or derivatives thereof, e.g. Lys, Arg
C07K 5/107 - Tetrapeptides the side chain of the first amino acid containing carbocyclic rings, e.g. Phe, Tyr
05 - Pharmaceutical, veterinary and sanitary products
42 - Scientific, technological and industrial services, research and design
Goods & Services
Biopharmaceutical preparations for the prevention and treatment of mitochondrial, cardiovascular, renal, ophthalmic, neurologic, myopathic and metabolic diseases and disorders; biological preparations for the prevention and treatment of cellular abnormalities Scientific research and development; biopharmaceutical research, development and testing; medical and scientific research in the mitochondrial, cardiovascular, renal, ophthalmic, neurologic, myopathic and metabolic system fields
05 - Pharmaceutical, veterinary and sanitary products
42 - Scientific, technological and industrial services, research and design
Goods & Services
Biopharmaceutical preparations for the prevention and treatment of mitochondrial, cardiovascular, renal, ophthalmic, neurologic, myopathic and metabolic diseases and disorders; biological preparations for the prevention and treatment of cellular abnormalities Scientific research and development; biopharmaceutical research, development and testing; medical and scientific research in the mitochondrial, cardiovascular, renal, ophthalmic, neurologic, myopathic and metabolic system fields
95.
Crystalline bis- and tris-hydrochloride salt of elamipretide
C07K 5/00 - Peptides having up to four amino acids in a fully defined sequenceDerivatives thereof
C07K 5/11 - Tetrapeptides the side chain of the first amino acid containing more amino groups than carboxyl groups, or derivatives thereof, e.g. Lys, Arg
C07K 5/09 - Tripeptides the side chain of the first amino acid containing more amino groups than carboxyl groups, or derivatives thereof, e.g. Lys, Arg
The disclosure provides compositions and methods relating to aromatic-cationic peptides. The methods comprise administering to the subject an effective amount of an aromatic-cationic peptide to subjects in need thereof. For example, the peptides may be administered to subjects in need of a mitochondrial-targeted antioxidant.
C07C 229/26 - Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having more than one amino group bound to the carbon skeleton, e.g. lysine
C07K 5/065 - Dipeptides the side chain of the first amino acid containing carbocyclic rings, e.g. Phe, Tyr
C07K 5/068 - Dipeptides the side chain of the first amino acid containing more amino groups than carboxyl groups, or derivatives thereof, e.g. Lys, Arg
C07K 5/072 - Dipeptides the side chain of the first amino acid containing more carboxyl groups than amino groups, or derivatives thereof, e.g. Asp, Glu, Asn
C07K 5/087 - Tripeptides the side chain of the first amino acid containing carbocyclic rings, e.g. Phe, Tyr
C07K 5/09 - Tripeptides the side chain of the first amino acid containing more amino groups than carboxyl groups, or derivatives thereof, e.g. Lys, Arg
C07K 5/107 - Tetrapeptides the side chain of the first amino acid containing carbocyclic rings, e.g. Phe, Tyr
C07K 5/11 - Tetrapeptides the side chain of the first amino acid containing more amino groups than carboxyl groups, or derivatives thereof, e.g. Lys, Arg
G01N 33/68 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing involving proteins, peptides or amino acids
98.
Methods of identifying and monitoring mitochondrial dysfunction using monocyte screening
The present technology provides methods for detecting and diagnosing diseases and conditions characterized by mitochondrial dysfunction using monocytes as an indicator of the dysfunction.
G01N 33/50 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing
99.
Compositions and methods for the treatment of amyotrophic lateral sclerosis, Parkinson's disease, Parkinson's disease with dementia, dementia with Lewy bodies, and multiple system atrophy
The present disclosure provides novel methods for treating or preventing amyotrophic lateral sclerosis (ALS), methods for delaying the onset of neurological symptoms associated with ALS, increasing survival in subjects afflicted with ALS, and attenuating the decline of muscle strength associated with ALS in a subject in need thereof. The present disclosure also provides methods for treating or preventing α-synucleinopathy or TDP-43 proteinopathy. The methods comprise administering to the subject an effective amount of a mitochondria-targeting peptidomimetic compound, such as (R)-2-amino-N—((S)-1-(((S)-5-amino-1-(3-benzyl-1,2,4-oxadiazol-5-yl)pentyl)amino)-3-(4-hydroxy-2,6-dimethylphenyl)-1-oxopropan-2-yl)-5-guanidinopentanamide, or a pharmaceutically acceptable salt, stereoisomer, tautomer, hydrate, and/or solvate thereof.
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
A61K 31/135 - Amines, e.g. amantadine having aromatic rings, e.g. methadone
A61K 31/197 - Carboxylic acids, e.g. valproic acid having an amino group the amino and the carboxyl groups being attached to the same acyclic carbon chain, e.g. gamma-aminobutyric acid [GABA], beta-alanine, epsilon-aminocaproic acid or pantothenic acid
A61K 31/4152 - 1,2-Diazoles having oxo groups directly attached to the heterocyclic ring, e.g. antipyrine, phenylbutazone, sulfinpyrazone
A61K 31/4178 - 1,3-Diazoles not condensed and containing further heterocyclic rings, e.g. pilocarpine, nitrofurantoin
A61K 31/4184 - 1,3-Diazoles condensed with carbocyclic rings, e.g. benzimidazoles
The present technology is directed to compositions and methods for preventing, ameliorating, or reducing the severity of one or more signs, or symptoms associated with a reduction of function, decreased expression level of, and/or deficiency in one or more of COL4A3, COL4A4 and COL4A5 genes. Also disclosed herein are methods of preventing or treating Alport Syndrome in a mammalian subject, reducing risk factors associated with Alport Syndrome, and/or reducing the likelihood or severity of Alport Syndrome. The methods comprise administering to the subject an effective amount of an aromatic-cationic peptide.
